Treatment for Pompe disease now available for Belgian patients

The Belgian Official Gazette reports that Minister for Public Health Rudy Demotte has approved the complete reimbursement of Myozyme™ as of 1 May 2007. Myozyme™ is a medicine used in treating Pompe disease, a progressive and often life-threatening inherited muscular disorder that affects fewer than 10,000 people worldwide. There are fewer than 30 known patients with Pompe disease in Belgium at this moment. Myozyme™ is the first treatment ever approved for Pompe disease and the first for an inherited muscle disorder.

“For the Belgian patients with Pompe disease and their family members, this is a historical moment,” says Hilde Stoop, Managing Director of Genzyme Belgium, the biopharmaceutical company that developed Myozyme™ and that will be manufacturing it at its production unit in Geel (Belgium). “The complete reimbursement for this treatment is very hopeful and it is a big step forward for all patients suffering from Pompe disease. This announcement offers new perspectives for the future as well as assurance to all Pompe disease patients that this life saving treatment will be accessible to them.”

Pompe disease expresses itself in many different clinical symptoms, but one characteristic of it is that all patients suffer from increasing muscle weakness and/or respiratory complications. However, the degree to which the disease progresses varies widely from patient to patient. Newborn babies with the disease develop a massively enlarged heart a few months after birth and, due to the strongly decreased muscle tone, do not develop normally. The majority of these young patients die from muscular complications and failure of vital functions before they reach their first birthday.

Depending on whether the symptoms appear during childhood, puberty or adulthood, they vary considerably in seriousness and speed of progression. Most patients eventually become wheelchair bound or are in need of respiratory help. As a rule, there is a risk among these patients of earlier death due to respiratory failure.

Myozyme™ was approved on 29 March 2006 by the European Medicines Agency (EMEA) for the treatment of patients with Pompe disease.